Enabling Regenerative Medicine Disruptive Stem Cell Technology
Enabling Regenerative Medicine
Disruptive Stem Cell Technology
This presentation includes statements that are, or may be deemed, ‘‘forward-looking statements.” In some cases, these forward-looking statements can be identified by the use of forward-looking terminology, including the terms “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately,” potential” or, in each case,their negative or other variations thereon or comparable terminology, although not all forward-looking statements contain these words. For example, forward-looking statements are used inthis presentation when we discuss the timing of our clinical trials, the potential for the Company’s technology to be used for various indications and applications, the potential advantages ofthe Company’s technology over its competitors, our future plans which may include licensing and our expected timeline for regulatory approval of our products and studies. We have basedthese forward‐looking statements on assumptions and assessments made by our management in light of their experience and their perception of historical trends, current conditions,expected future developments, and other factors they believe to be appropriate. Because such statements deal with future events and are based on our current expectations, they are subjectto various risks and uncertainties and actual results, performance or achievements could differ materially from those described in or implied by the statements in this presentation. In addition,historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to hereinwould be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in theforward-looking statements: our expectations regarding the timing of commencing clinical trials with respect to our ApoGraft process and our Apotainer selection kit; our expectationsregarding the progress of our clinical trials, including the duration, cost and whether such trials will be conducted at all; our intention to hold meetings with regulators and apply for regulatoryapproval for our product candidates, and the costs and timing of such regulatory approvals; the likelihood of regulatory approvals for our product candidates; the timing and cost of thedevelopments of our prototype Apotainer selection kit; our expectation to obtain a sufficient supply of FasL for our needs in the foreseeable future; the market size and future sales of ourproduct candidates or any other future products or product candidates; that our technology may potentially improve the safety and efficacy of regenerative medicine stem cell therapy andother potential advantages of our selection process for physicians, academics, researchers and others; our intention to expand our product development and build a diversified productportfolio of Apograft products for a broad spectrum of market segments; and our estimates regarding anticipated expenses, capital requirements and our needs for substantial additionalfinancing. By their nature, forward-looking statements involve risks and uncertainties because they relate to events, competitive dynamics, and healthcare, regulatory and scientificdevelopments and depend on the economic circumstances that may or may not occur in the future or may occur on longer or shorter timelines than anticipated. Although we believe that wehave a reasonable basis for each forward-looking statement contained in this presentation, we caution you that forward-looking statements are not guarantees of future performance and thatour actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements containedin this presentation as a result of, among other factors, the factors referenced in the “Risk Factors” section of contained in our most recent Annual Report on Form 20-F filed with theSecurities and Exchange. In addition, even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with theforward-looking statements contained in this presentation, they may not be predictive of results or developments in future periods. Any forward-looking statement that we make in thispresentation speaks only as of the date of such statement, and we undertake no obligation to update such statements to reflect events or circumstances after the date of this presentation.More detailed information about the risks and uncertainties affecting the Company is contained under the heading “Risk Factors” in the Annual Report and in the Company’s period filings withthe SEC.
Forward-Looking Statements
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Our Mission
Cellect aims at facilitating Stem Cell technologies to
reach the holy grail of medicine:
• stop fixing by chemicals, poisons, radiation and
massive surgery, and instead
• replace the damaged tissues and organs using
revolutionary Stem Cell technologies.
Cellect offers an enabling platform for Stem Cell
“cellection” and cost-effective mass production of
high quality Stem Cells.
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The Challenge • For Stem Cells to be used for regenerative
medicine, there is a need for large amounts of
Enriched and High Quality Stem Cells.
• Many companies with Stem Cell products
encounter challenges at large scale
manufacturing, leading to
- failures at later stage clinical trials and/or
- unacceptably expensive products.
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Our Vision• Develop Cellect-owned safe, and cost-effective
products for specific Stem Cell treatments
• Propose Stem Cell companies an efficient, cost
effective and standardized selection process for Stem
Cells, enabling reproducible mass production of raw
material from any source for any indication.
“STEM CELL INDUSTRY INFLECTION POINT IS STANDARDIZATION AND CELLECT HAS THE SOLUTION” (Ray Dirks)http://www.raydirks.com/2017/10/18/stem-cell-industry-inflection-point-is-standardization-and-cellect-has-the-solution/
Technology
• Cellect develops a technology enabling mass production of Stem Cells for pharma,
medical and research uses
• Strong IP portfolio with 7 patent families; 4 of which already granted in US
• Phase I/II clinical trial: First cohort successfully completed one month– no related adverse
effects
Strategy
• Does not compete with Stem Cell companies, but proposes enabling and standardization
of more effective, safer and less costly transplant products manufacturing.
• Seeking to enter into multiple licensing deals - initial revenues expected already in 2018
Management
• Seasoned professional management team
• KOLs on BOD and advisory board
Healthy Finances
• Sufficient cash to support current Phase I/II clinical trial and pre-clinical pipeline
development
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Cellect –Investment OverviewNASDAQ:APOP
Program I: Hematopoietic Stem Cells (HSCs) Bone Marrow Transplantations
Challenge Existing Product
and Process
Trade Off
(selection)
Cellect’s Solution – ApoGraftTM Technology
in the ApoTainerTM
Acute Graft vs Host
Disease (aGvHD)
in 30 - 50% of
patients
Stem Cell purification using
specific markers
- Low GvHD
- Failures of transplant
- Recurrence of cancer
(low GvL)
Design:
- Cellect’s ApoTainer comprising FasL induces cell death
(Apoptosis) of mature cells
- Results in significant GvHD reduction
- Full preservation of transplant
- Preserves anti-cancer activity (high GvL)
- Fast, safe and cost-effective
Non-toxic
&
ineffective
Effective
&
highly toxic
Cellect’s ApoTainerTM comprising
apoptosis-inducing molecules:
Negative Selection of GvHD-
inducing cells
• The clinical score measures the severity of GvHD at the indicated times in irradiated transplanted NSG mice.• Each symbol represents the mean value ± SEM for 10 mice in each group.• MPBC = human mobilized peripheral blood cells• ApoGraft – MBPCs after incubation according to ApoGraft protocol with indicated FasL concentration
Efficacy Study:ApoGraft Improves GvHD Clinical Score
0 2 0 4 0 6 0 8 0 1 0 0
0
2
4
6
8
T im e (d a y s )
Cli
nic
al
sc
ore
M P B C s c o n t r o l
A p o G r a f t 2 5 n g / m l F a s L
A p o G r a f t 1 0 0 n g / m l F a s L
**
***
• Severe GvHD in mice injected with human
MBPCs, including high mortality
• Negligible GvHD detected with human
MBPCs injected after ApoGraft process
Cellect’s ApoTainerTM vs. Alternatives
Current Procedures ApoTainerTM **
Risk of GvHD 30% - 50% Negligible
Chemotherapy Wide Reduced
Rate of Infection High Low
Procedure and
hospitalizationMonths Days
Cell Selection Cost ~ $70,000 < $10,000
Total cost of procedure ~ $300,000 < $100,000
** Company Estimates
• 2 hours simple bed-side procedure• No major change in SOC
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Program II: Mesenchymal Stem Cells (MSCs)Broad Applications in Stem Cell Therapies
Challenges Existing
Process
Trade Off
(expansion)
Cellect’s Solution - ApoGraftTM
Technology in the ApoTainerTM
- Fat-derived MSCs (ASCs)
expansion lengthy
- Major cell source for esthetic,
orthopedic and other indications
(> 500 non - regulated clinics in
USA) with recurrent failures due to
lack of standardization
- Few approved products –
challenging prices
- No selection
- Lengthy expansion
time (weeks)
- High cell numbers, but
also high cost
- Often loss of
“stemness” and
therefore reduced
efficacy
Design:
- Results in significantly higher cell numbers with
enhanced “stemness” in early passages
- Shorter expansion times
- Safe and cost-effective production
High
“stemness”
&
low yield
Low
“stemness”
&
high yield
Cellect’s ApoTainerTM
comprising apoptosis-inducing
molecules: death of mature
cells – Positive Selection of
Stem Cells
Adipose Stem Cells (ASCs) – Expansion with FasL
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• FasL selects EARLY progenitor cells
• FasL induces proliferation of ASC
0
0.1
0.2
0.3
0.4
0.5
Control Fas-L
Ce
ll N
o. x
10
6
ASC Expansion
Large colony Small colony
Early
progenitor cellsprogenitor cells
0
10
20
30
40
50
60
Control Fas-L
No
. of
larg
e c
olo
nie
s
CFU-F Assay of Stem Cell Progenitor Cells
Dual effect drives improved cost-benefit for product manufacturing
Cellect in The Stem Cells Value Chain
Raw
Material
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Highly effective
selection -
Reduced cost
Positive
Selection
HSC
MSC
Negative
Selection
Time Cost
Reduced
GvHD
Safety
Highly effective
expansion-
Reduced cost
Standardization
Efficacy
Quality
~2 hours
No change
in SOC
Few days
Shorter
Stem Cells Enabling Technology – Fast Growing Market Potential
Yen-Shun et al. Int J Stem Cell Res Ther 2016, 3:029
Mesenchymal Stem Cells –
Clinical Trials
• Number of clinical trials with MSCs fast growing
• Large number of possible indications
• Largest number of trials for GvHD, Heart
Disease and Orthopedics, fields with Cellect
involvement
• All procedures starting with similar MSCs, all
potential candidates for Cellection
Hematopoietic Stem Cells –
Global Transplantation Market
• Fast growing market with estimated WW CAGR of
almost 12.8% until 2025 (TMR)
• Currently almost exclusive procedure in malignant
diseases
• Large number of potential non-malignant indications
(severe auto-immune diseases, metabolic diseases,
immune deficiencies etc.)
• Main challenge limiting outcome and broader use of
HSCT: cytotoxicity - GVHD12
Scalable Versatile Product Platform
Research Clinic Industry
• Combination product (biological and device)
• From test tube to industrial-level bioreactor size
Business Model & Strategy• Medical proof of concept – launch a consumable medical device with first indication –
substantially reduce GvHD
• Build a brand of quality (“Cellected”) to establish leadership position
• Leverage growing IP portfolio (7 patent families)
• Planned additional internal programs – development to product launch:
o POC indications
o in-licensing
• Technology Platform – Enter into strategic deals with pharma, biotech, research and
biobanking
o out licensing: upfront + milestone payments + royalties
o spin-offs
• Research collaborations for other cell selection technologies14
Clinical Program
2016 2017 2018 2019 2020
Ph I/II GvHD (ISR)
ApoTainer Optimization
Non-interventional
MSC Preclinical
GvHD US study
ApoTainer Pivotal Study
Ph I/II MSC
ApoGraft
ApoTainer
MSC
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ApoTainer Comparability
Ph I/II GvHD (ISR) Follow-up Study
ApoGraft Phase I/II Clinical Trial
Rambam Medical Center Haifa (Israel)
Hadassah Medical Center Jerusalem (Israel)2 sites
An Open-Label Phase I/II, Pilot, Staggered Four-Cohort Safety and Proof-of-Concept Study
of ApoGraft in the Prevention of Acute Graft Versus Host Disease Trial
12 matched related donor-recipient pairsTrial Group
Safety and preliminary efficacyTrial Purpose
180 daysDuration
Three patients (first cohort) completed 1 month follow up
- full engraftment and no related adverse effects
- dose escalation approved by DSMB
- patient enrollment continues
Current Status
(Jan 2018)
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Financial Data
• Solid balance sheet with $~9.5 M of cash as of 9/30/2017; $~4.0 M raised in Jan 2018; no loans
• Sufficient cash to support current Phase I/II clinical trial and pre-clinical pipeline development
• Ordinary shares outstanding - ~130 million (equivalent to ~6.5 million ADSs)
• IPO on NASDAQ in 2016. De-listed from the TASE (Sep 2017)
Average Volume (*)
* Based on 90 days average
ADSs US$
NASDAQ 115,620 ~ 897,200~
TASE – De-listed 0 0
Total 115,620 ~ 897,200~
(*) Holdings and Average volume as of February 9th, 2018
Founders, 33.99%
>10% Investor, 11.05%
Public, 54.96%
Holdings (*)
Experienced Management Team / AdvisorsDr. Shai Yarkoni MD, PhD, Co- Founder & CEOSenior executive in the biotechnology industry. Founder and CEO of Target-In Ltd., a cancer biotech therapeutics company. Founded and, until recently, managed Bio-Negev.
Eyal Leibovitz, CFOMore than 25 years of experience in senior management, finance, IR, M&A and business development in international companies. Mr. Leibovitz has served as CFO of Evogene, N-trig Ltd., Kamada Ltd., and New Media Communications.
Dr. Ronit Bakimer-Kleiner PhD, Chief Development OfficerMore then 20 years of experience as a scientist and executive manager in the biotechnological industrial environment, has amassed comprehensive knowledge in cell therapy and GMP manufacturing and has an extensive background in immunology, autoimmunity and cell biology, and in management of GMP facilities and tech-transfer.
Dr. Tamara Byk-Tennenbaum, PhD, MBA, VP OperationsMore than 25 years experience of scientific management in the fields of hematopoietic stem cells, hemostasis, neurobiology and gene therapy. Served as VP Technology Platform at Evogene, R&D Associate Director at Omrix (Ethicon Biosurgery - JnJ) and Scientific Director at Teva. Extensive experience in product development from early stages to launch, including scale up and GMP manufacturing.
BOARDsKasbian Nuriel Chirich, Co-Founder & Chairman of the Board Businessman with extensive financial and business expertise. Leads several business ventures in East Africa and Israel and is the Honorary Consul of Tanzania in Israel.
Dr. Michael Berelowitz MD, Board MemberPreviously Sr. VP, Head of Clinical Development and Medical Affairs, Pfizer Specialty Care Business Unit and Head of Global Medical at Pfizer.
David Braun, Board MemberHead of Merck Group’s Medical Device Business
Dr. Susan Alpert, Advisory BoardPreviously Head of Medical Device division at the FDA and EVP of Regulatory Affairs at both Medtronic (NYSE:MDT) and CR Bard (NASDAQ:BCR).
Dr. Corey Cutler MD, Advisory Board Head of bone marrow transplantation at world-renowned Dana-Farber cancer institute, an Associate Professor of Medicine at Harvard Medical School.
Prof. John F. DiPersioDeputy Director, Alvin J. Siteman Cancer Center and Chief of the Division of Oncology at Washington University School of Medicine in St. Louis and the Virginia E. and Samuel J. Golman Professor of Medicine.
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Strong IP Protection 7 families of global patent applications (4 already issued)
• Patent for the concept of using apoptosis-inducing agents for Stem Cells selection - issued
• Patents for composition of matter of ApoTainers™- issued
• Patents covering methods of use - issued
• Patents covering method of manufacturing ApoTainers™
• Patent for Mesenchymal Stem Cells selection
• Strong IP validated by proof-of-concept studies
• Expiration of concept patent - 2029
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SummaryNASDAQ:APOP
• Cellect technology aims to enable standardized mass production of Stem Cells for
research, clinics and pharma.
• Using the transformational Cellect Platform, Stem Cell products are intended to be safer
and more cost effective.
• Cellect platform uses are expected to drive initial revenues already in 2018
• Strong IP portfolio with 7 patent families; 4 of which already granted in US
• US orphan drug designation granted (2017)
• Clinical trial Phase I/II started and first cohort completed one month with no related
adverse effects (Jan 2018). Dose escalation approved by DSMB.
• Seasoned management team and Advisory Board
• Healthy finances
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Thank You
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